Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
Information source: Shire
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Gaucher Disease
Intervention: velaglucerase alfa (Biological)
Phase: Phase 3
Status: Completed
Sponsored by: Shire Official(s) and/or principal investigator(s): Bjorn Mellgard, M.D., Study Director, Affiliation: Shire
Summary
Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB)
that leads to progressive accumulation of glucocerebroside within macrophages and subsequent
tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. The
disease has been classified into 3 clinical subtypes based on the presence or absence of
neurological symptoms and severity of neurological disease. Type 1 Gaucher disease affects
an estimated 30,000 persons worldwide and is the most common. Type 1 Gaucher disease does
not involve the central nervous system. Patients with type 2 Gaucher disease present with
acute neurological deterioration, which leads to early death. Those with type 3 disease
typically display a more sub-acute neurological course, with later onset and slower
progression.
The primary objective of this study is to evaluate the safety of every other week dosing of
velaglucerase alfa in Japanese patients with Gaucher disease.
Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type
1 Gaucher disease.
Clinical Details
Official title: A Multicenter, Open-Label Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Number of Severe Adverse Events (SAE)Number of Treatment Emergent Adverse Events (TEAE) Development of Anti-velaglucerase Alfa Antibody Number of Infusion- Related Adverse Events Number of Patients With Concomitant Medication
Secondary outcome: Change From Baseline in Hemoglobin ConcentrationChange From Baseline in Platelet Count Change From Baseline in Liver Volume, Normalized to Body Weight Change From Baseline in Spleen Volume, Normalized to Body Weight Change From Baseline in Plasma Chitotriosidase Levels Change From Baseline in CCL18 Levels
Detailed description:
Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB)
that leads to progressive accumulation of glucocerebroside within macrophages and subsequent
tissue and organ damage; typically of the liver, spleen, bone marrow, and brain.
Gaucher disease has been designated in the list of Specified Rare and Intractable Diseases
by Specified Disease Treatment Research Program of Ministry of Health, Labor and Welfare
(MHLW) as one of "lysosomal storage diseases" since 2001. Gaucher disease is also
designated in the Medical Aid Program for Specified Categories of Chronic Pediatric
Diseases.
The prevalence of mutations and the phenotype of patients with Gaucher disease in Japan
differs from that in non-Japanese populations. Some patients with type 1 Gaucher disease in
Japan have more severe and progressive disease compared to non-Japanese patients and the
disease is characterized by an earlier onset of symptoms.
Velaglucerase alfa, a highly-purified form of the naturally occurring enzyme
glucocerebrosidase, has been developed as an enzyme replacement therapy for Gaucher disease
for the symptoms (anemia, thrombocytopenia, hepatomegaly, splenomegaly, and bone
manifestation).
The primary objective of this study is to evaluate the safety of every other week dosing of
velaglucerase alfa in Japanese patients (naive or previously treated with imiglucerase) 2
years of age and older with Gaucher disease.
Eligibility
Minimum age: 2 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- The patient has a documented diagnosis of Gaucher disease
- The patient is at least 2 years of age
- Female patients of child bearing potential must agree to use a medically acceptable
method of contraception at all times during the study
- The patient, the patient's parent(s) or legal guardian(s) has provided written
informed consent that has been approved by the Institutional Review Board/Independent
Ethics Committee (IRB/IEC)
- The patient must be sufficiently cooperative to participate in this clinical study as
judged by the Investigator
Patients who are switched from imiglucerase ERT must meet the following additional
criteria:
- Received treatment with imiglucerase for a minimum of 12 consecutive months
- Meet predefined limits for hemoglobin concentration and platelet counts
Patients naïve to treatment for Gaucher disease must meet the following additional
criteria:
- Not received treatment for Gaucher disease (investigational or approved products)
within 12 months prior to study entry
- Have Gaucher disease related anemia and at least one of the following: moderate
splenomegaly or, Gaucher disease-related thrombocytopenia or Gaucher disease-related
enlarged liver
Exclusion Criteria:
- Treatment with any investigational drug or device within the 30 days prior to study
entry (time of informed consent); such use during the study is not permitted
- Positive for hepatitis B or hepatitis C.
- Non-Gaucher disease related anemia
- The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to
understand the nature, scope, and possible consequences of the study
- Significant comorbidity, as determined by the Investigator that might affect study
data or confound the study results
- The patient is unable to comply with the protocol or is unlikely to complete the
study, as determined by the Investigator
- The patient has experienced a severe (grade 3 or higher) infusion-related
hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any ERT
(approved or investigational)
- Currently receiving red blood cell growth factor, (eg, erythropoietin) or chronic
systemic corticosteroids in the last 6 months
- Patient has had a splenectomy or the patient has an active, clinically significant
spleen infarction within 12 months of screening
- Patient has worsening bone necrosis within 12 months of screening
- The patient is pregnant or lactating.
Locations and Contacts
Osaka City University Hospital, Osaka 545-0051, Japan
Hamamatsu University School of Medicine, Hamamatsu, Shizuoka 431-3192, Japan
The Jikei University School of Medicine, Minato-ku, Toyko 105-8461, Japan
Additional Information
Starting date: March 2012
Last updated: July 8, 2014
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